Stem Cells Therapy in Degenerative Diseases of the Retina

Phase 1

• Conditions: Age Related Macular Degeneration; Retinal Degeneration; Retinitis Pigmentosa; Stargardt Disease 1
• Interventions: Biological: Stem/progenitor cells transplantation

• Registration Number: NCT03772938
• Lead Sponsor: Pomeranian Medical University Szczecin

Brief Summary

The purpose of this study is to test the safety and effectiveness of an autologous bone marrow-derived stem/progenitor cells administered intravitreously in the subjects with degenerative diseases of the retina.

Detailed Description

Degenerative diseases of the retina are challenging for ophthalmologists. This is a common term that covers heterogenous group of diseases, f.e. retinitis pigmentosa, Stargardt disease, Best's disease or age related macular degeneration. Undetermined etiology, wide range of factors that may trigger the onset of the disease and modulate it's course impede the implementation of an effective treatment. Currently, a stem cells therapy seems to be promising option in patients with degenerative diseases of the retina. The purpose of this prospective, nonrandomized, open label, pilot study is to conduct the investigation of the safety and efficacy of intravitreal injection of autologous bone marrow-isolated stem/progenitor cells with different selected phenotypes into the subjects with degenerative diseases of the retina. Especially, this clinical trial is designated to test the therapeutic (pro-regenerative and neuro-protective) functions of different stem/progenitor cell populations able to secrete bioactive neurotrophic factors. All patients enrolled will have a documented history of degenerative disease of the retina prior to study enrollment. Next, autologous bone marrow-isolated stem/progenitor cells intravitreous injection will be performed. Finally, treatment safety, adverse events and exploratory parameters, including best corrected visual acuity, visual field and electroretinography parameters, to establish disease progression rate will be recorded throughout the duration of the post-treatment follow up period.

Study Timeline

• Study First Submitted: 2018-11-03
• Status Verified: 2018-12
• Study First Submitted QC: 2018-12-08
• Study First Posted: 2018-12-12
• Study Start: 2018-12-13
• Last Update Submitted: 2018-12-14
• Last Update Posted: 2018-12-17
• Primary Completion: 2020-02-28
• Study Completion: 2020-03-31

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

• diagnosed degenerative disease of the retina,
• age 18-65 years,
• best corrected visual acuity max. 0,2 (Snellen letter chart),
• good understanding of the protocol and willingness to consent,
• signed informed consent.

Exclusion Criteria

• concomitant eye disease (glaucoma, etc.)
• concomitant of other systemic disease or diseases,
• inflammation (high protein or lymphocytosis in the CSF), active infections.
• diabetes,
• cardio-vascular disorders,
• cancer,
• autoimmune diseases,
• renal failure,
• impaired hepatic function,
• subject unwilling or unable to comply with the requirements of the protocol,
• patient has been treated previously with any cellular therapy,

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Stem/progenitor cells transplantation	Stem/progenitor cells transplantation	Intervention: A single intravitreal injection of autologous bone marrow-derived stem/progenitor cells will be performed.
Standard treatment of degenerative disease of retina	Stem/progenitor cells transplantation	Symptomatic treatment of degenerative disease of retina without biologic cell-based treatment

Primary Outcome Measures

Name	Time	Method
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0	12 months	Confirm the safety of autologous bone marrow stem/progenitor cell intravitreal injection in enrolled patients by repeated follow-up over one year with clinical evaluations.

Secondary Outcome Measures

Name	Time	Method
Efficacy of autologous bone marrow stem/progenitor intravitreal injection in enrolled patients.	12 months	Best corrected visual acuity, ETDRS chart \[number of letters\]
Intraocular pressure	12 months	Pascal tonometer \[mmHg\]
Optic disk retinal nerve fiber layer	12 months	optical coherence tomography \[um\]
Central macular thickness	12 months	optical coherence tomography \[um\]
Ganglion cell complex thickness	12 months	optical coherence tomography \[um\]
Choroidal thickness	12 months	Enhanced depth imaging optical coherence tomography \[um\]
Choroidal volume	12 months	Enhanced depth imaging optical coherence tomography \[mm3\]
Computed perimetry (30-2 and 10-2 module)	12 months	mean deviation, pattern standard deviation \[B\]
Goldmann perimetry with color filters	12 months	\[degrees\]
Contrast sensitivity	12 months	Pelli-Robson chart \[number of letters\]
Function of the photoreceptors (rods and cones), inner retinal cells (bipolar and amacrine cells), and ganglion cells.	12 months	Electroretinography (ERG) examination: * amplitude of a and b waves \[V\],; * culmination time of a and b waves \[s\],; * culmination time of q1-q3 waves \[s\].
Function of the photoreceptors	12 months	Multifocal electroretinography (mfERG) examination: * retinal response density \[V/degree 2\],; * culmination time of P1 wave in 6 rings \[s\].
Function of ganglion cells	12 months	Pattern electroretinography (PERG) examination: * amplitude of P50 and N95 waves \[V\],; * culmination time of P50 wave \[s\].

Trial Locations

Locations (1)

I Department of Ophthalmology; 🇵🇱 Szczecin, Poland